ASCENDIS PHARMA A/S TransCon CNP is an investigational long-acting prodrug of C-type natriuretic peptide (CNP), designed to provide continuous exposure of CNP at safe, therapeutic levels via a single, weekly subcutaneous dose.
The ACcomplisH Trial evaluated 57 children with achondroplasia aged 2 to 10 years old, randomized in a 3:1 ratio to receive either sequential ascending doses of once-weekly TransCon CNP or placebo for 52 weeks. All 57 randomized children completed the blinded portion of ACcomplisH and are currently continuing in the open label extension (OLE) at the 100 -microg/kg/week dose.
The trial met its primary objectives, demonstrating that TransCon CNP at 100 -microg/kg/week was superior to placebo on the primary efficacy endpoint of AHV at 52 weeks. A slide presentation with these data can be found on the Investor Relations & News section of the
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Forward-Looking Statements
This press release contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, included in this press release regarding Ascendis' future operations, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to (i) TransCon CNP's ability to provide continuous exposure of CNP at safe, therapeutic levels via a single, weekly subcutaneous dose, (ii) TransCon CNP's potential to meet patient and caregiver needs for a safe, effective, tolerable and convenient treatment and to become an accepted and preferred treatment method, (iii) Ascendis' plans to advance TransCon CNP in its new Phase 2b ApproaCH trial and to bring TransCon CNP to patients as fast as possible, (iv) Ascendis' use of its TransCon technologies and platform to create new and potentially best-in-class therapies and build a leading, fully integrated, global biopharma company and (v) Ascendis' use of its TransCon technologies to create new and potentially best-in-class therapies. Ascendis may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions, expectations and projections disclosed in the forward-looking statements. Various important factors could cause actual results or events to differ materially from the forward-looking statements that Ascendis makes, including the following: dependence on third party manufacturers, distributors and service providers for Ascendis products and product candidates; unforeseen safety or efficacy results in its development programs or on-market products; unforeseen expenses related to commercialization of any approved Ascendis products; expenses related to Ascendis' development programs; unforeseen selling, general and administrative expenses, other research and development expenses and Ascendis' business generally; delays in the development of its programs related to manufacturing, regulatory requirements, speed of patient recruitment or other unforeseen delays; Ascendis' ability to obtain additional funding, if needed, to support its business activities; the impact of international economic, political, legal, compliance, social and business factors, including inflation, and the effects on its business from the worldwide COVID-19 pandemic and the ongoing conflict in the region surrounding
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